A one-year, open-label, single arm, multi-center trial evaluating the efficacy and safety of oral ICL670 (20 mg/kg/day) in patients diagnosed with transfusion-dependent iron overload
- Conditions
- Transfusional iron overload
- Registration Number
- EUCTR2004-003953-16-DK
- Lead Sponsor
- ovartis Pharma Services AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 1541
• patients presenting with transfusion-related iron overload (independent of underlying condition) with transfusional iron overload as shown by a serum ferritin level of = 1000 ng/ml, or with serum ferritin < 1000 ng/ml, but with history of multiple transfusions (>20 transfusions or 100 ml/kg of packed red blood cells) and LIC > 2 mg Fe/g dw (as confirmed by R2-MRI).
•age = 2 years
• Adult patients: written informed consent
• Pediatric patients: written informed consent by their parents or legal guardians on the patient’s behalf in accordance with the national legislation. If capable, all patients should also personally sign their written informed assent.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
• non-transfusion-related hemosiderosis
• clinical evidence supporting the need of intensive chelation
• mean levels of alanine aminotransferase (ALT) > 300 U/l
• uncontrolled systemic hypertension
• serum creatinine above the upper limit of normal (ULN)
• history of nephrotic syndrome
• history of clinically relevant ocular toxicity related to iron chelation
• systemic diseases which would prevent the patient from undergoing study treatment
• psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment
• pregnant or breastfeeding patients
• treatment with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days
• any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug
• history or likelihood of non-compliance to medical regimens
• history of drug or alcohol abuse within the past 12 months or evidence of such abuse during the run-in period
• positive test to HIV
•life expectancy of < 1year
• pediatric patients only: body weight which prevents the use of the smallest tablet strength (125 mg) for proper dosing
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method